Ruconest (Santarus/Pharming) is re-filed with FDA for treatment of Hereditary Angioedema

Santarus, Inc. and Pharming Group NV have announced the submission of a Biologics License Application (BLA) to the FDA to obtain marketing approval for Ruconest (recombinant human C1 esterase inhibitor) 50 U/kg, an investigational drug for the treatment of acute angioedema attacks in patients with Hereditary Angioedema (HAE). The safety and efficacy of Ruconest for the treatment of HAE attacks were evaluated in a clinical program that included a Phase III randomized placebo-controlled study conducted under a Special Protocol Assessment agreement with the FDA. The pivotal Phase III clinical study showed statistically significant and clinically relevant improvement in the primary endpoint of time to beginning of relief of symptoms for Ruconest compared with placebo. The Ruconest clinical program also included two additional randomized placebo-controlled studies and four open label treatment studies. In total, the BLA dossier includes ten clinical studies covering 940 administrations in 236 subjects.

Santarus licensed certain exclusive rights from Pharming to commercialize Ruconest in North America for the treatment of acute attacks of HAE as well as other potential future indications.

Ruconest was EU approved for Swedish Orphan Biovitrium, in 2010, to treat attacks of hereditary angioedema in adults (aged 18 years or over).